To develop a breakthrough treatment for LGMD2B
Our research focuses on personalized stem cell therapy for LGMD2B using advanced gene-editing technologies.
What is LGMD2B?
LGMD2B, also known as dysferlinopathy, is a rare genetic muscle disorder caused by mutations in the DYSF gene. It leads to progressive muscle weakness and wasting, primarily affecting the muscles of the hips, shoulders, and limbs. Symptoms often begin in late adolescence or early adulthood, and the condition gradually worsens over time. LGMD2B is inherited in an autosomal recessive pattern.
Why is LGMD2B first?
The founder of this company is a patient with LGMD2B, as are his siblings. His firsthand experience as a patient and as a brother of other patients drives his deep commitment to advancing research in this field. He is also personally contributing to the research by providing his fibroblasts sample for the generation of stem cells, and by participating as a volunteer patient.
How to cure LGMD2B.
There have been numerous efforts to develop treatments to cure or alleviate the symptoms of LGMD2B. However, no significant solution has yet been established for this rare disease.
Fortunately, with recent advancements in biotechnology—particularly in stem cell therapy and gene editing—and with the FDA’s regulatory support for individualized medicine for rare diseases, we have now been able to develop a promising new approach to treating LGMD2B.
More than LGMD2B.
We will build the process and platform based on the research results to cure other rare muscular disorders caused by gene mutations as well.